MALVERN, PA — Biopharmaceutical company Ocugen, Inc. (NASDAQ: OCGN) has announced the commencement of its Phase 1/2 GARDian clinical trial for OCU410ST (AAV5-hRORA), a gene therapy candidate being developed to treat Stargardt disease. The first patient in this trial was dosed last Friday.
Stargardt disease is a rare, genetically inherited disorder that causes retinal degeneration and progressive vision loss. It typically develops during childhood or adolescence and affects approximately 35,000 patients in the U.S.
Ocugen’s Chairman, CEO and Co-Founder, Dr. Shankar Musunuri, highlighted the significance of this trial, stating, “There is a significant unmet medical need for the approximate 35,000 patients in the U.S. living with Stargardt disease. It is critical to our mission to develop innovative treatments for inherited retinal diseases and this milestone is an important step in bringing our novel modifier gene therapies to people who desperately need them.”
The Phase 1/2 trial will assess the safety of unilateral subretinal administration of OCU410ST in subjects with Stargardt Disease. The trial will be conducted in two phases: Phase 1 is a multicenter, open-label, dose-ranging study, and Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study. Adult and pediatric subjects will be randomized in a 1:1:1 ratio to either one of two OCU410ST dose groups or to an untreated control group.
OCU410ST utilizes an Adeno-associated virus (AAV) delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. This represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptor (NHR) RORA that regulates pathway links to Stargardt disease such as lipofuscin formation, oxidative stress, compliment formation, inflammation, and cell survival networks.
Charles Wykoff, MD, PhD, Director of Research, Retina Consultants of Texas, expressed optimism about the trial, saying, “Initiation of this trial program investigating a new mechanism of action for the treatment of Stargardt disease is inspiring and brings hope to patients and families.”
This initiative by Ocugen marks an important step in the quest to develop effective treatments for inherited retinal diseases. It offers hope for patients suffering from Stargardt disease and could potentially form the basis for treating other similar conditions.
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